Clinical Efficacy and Safety of Fanhdi®, a Plasma-Derived VWF/Factor VIII Concentrate, in von Willebrand Disease in Spain: A Retrospective Study

Authors: Jiménez-Yuste, V; Alvarez-Román, MT; Palomo Bravo, Á; Galmes, BJ; Nieto Hernández, Maria Del Mar; Benítez Hidalgo, O; Marzo Alonso, C; Pérez González, NF; Coll, J; Núñez, R; Carrasco, M; García Candel, F; Gonzalez-Porras, JR; Hernández García, C; Varó Castro, MJ; Mir, R

Affiliations: Department of Hematology and Hemotherapy, Hospital Universitario La Paz, Autónoma University, Madrid, Spain; Hematology Service, Hospital Materno-Infantil de Málaga, Málaga, Spain; Hematology Service, Hospital Universitario Son Espases, Palma de Mallorca, Spain; Hematology and Hemotherapy Service, Complejo Universitario de Jaén, Jaén, Spain; Hematology and Hemotherapy Department, Hospital Vall d’Hebron, Barcelona, Spain; Hematology and Hemotherapy Service, Hospital Universitari Arnau de Vilanova, Lleida, Spain; Hematology and Hemotherapy Service, Complejo Universitario Torrecárdenas, Almería, Spain; Hematology Service, Complejo Hospitalario de Navarra, Pamplona, Spain; Hematology Service, Hospital Virgen del Rocío, Sevilla, Spain; Thrombosis and Hemostasis Unit, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain; Hematology and Hemotherapy Service, Hospital Clínico Universitario Virgen Arrixaca, Murcia, Spain; Department of Hematology, Hospital Universitario de Salamanca-IBSAL-USAL, Salamanca, Spain; Hematology and Hemotherapy Service, Hospital de Don Benito-Villanueva, Badajoz, Spain; Hematology Service, Hospital Universitario de Albacete, Albacete, Spain; Scientific and Medical Affairs Department, Grifols, Barcelona, Spain.

Publication: Clinical and applied thrombosis/hemostasis ; 2022 ; 28.

Abstract: OBJECTIVE: To evaluate the efficacy and safety of a plasma-derived factor VIII concentrate containing von Willebrand Factor (pdVWF/FVIII) in standard clinical practice in von Willebrand Disease (VWD) patients. METHODS: A retrospective, multicentric, observational study of VWD patients treated with Fanhdi®, a pdVWF/FVIII concentrate, from January 2011 to December 2017 was conducted at 14 centers in Spain. Efficacy and safety were evaluated for acute bleeding episodes, for prevention of bleeding in surgeries, and for secondary long-term prophylaxis. RESULTS: Seventy-two eligible patients, type 1, 2, 3 VWD (25%/38.9%/36.1%) were treated for spontaneous and traumatic bleeding (140 episodes, n = 41 patients), to prevent surgical bleeding (69 episodes, n = 43 patients); and for secondary long-term prophylaxis (18 programs, n = 13 patients). Replacement therapy with pdVWF/FVIII showed an excellent to good clinical efficacy in 96.7% of the bleeding episodes, 100% during surgical procedures and 100% during prophylaxis. No adverse events (AEs), nor serious AEs related to the product were observed. CONCLUSIONS: Fanhdi® was effective, safe and well tolerated in the management of bleeding episodes, the prevention of bleeding during surgeries, and for secondary long-term prophylaxis in VWD patients.